Volume 8, Issue 12

New Directions in CF Therapies

In this issue:

A truly effective cystic fibrosis (CF) disease modifying therapy needs to decrease the rate of lung function decline and improve both quality of life as well as life expectancy. The introduction and exceptional success of the CFTR protein potentiator ivacaftor began an exciting new era of progress in treating not just the symptoms of CF, but also the actual underlying mechanisms of the disease itself.

In this issue, Dr. Jennifer Taylor-Cousar from National Jewish Health and Dr. Shijing Jia from the University of Michigan analyze the current literature describing the newest frontiers of CF therapy.

Guest Faculty Disclosures

Dr. Taylor-Cousar has indicated that, as faculty at an institution that is part of the CF TDN, she has been a site PI on studies for Bayer, Celtaxys, Proteostasis, and Vertex Pharmaceuticals Incorporated. She has also received consulting fees from Gilead Sciences, Inc and Santhera. She has done clinical trial consulting and/or advisory boards for Gilead Sciences, Inc., Proteostasis, Protalix, Santhera, and Vertex Pharmaceuticals Incorporated. She has received grant funding from Gilead Sciences, Inc.

Dr. Jia has indicated that she has no financial interests or relationships with a commercial entity.

Unlabeled/Unapproved Uses

The authors have disclosed that they will be discussing the use of ivacaftor for rare mutations of CF. These methods have not been officially approved for this purpose.

Program Directors:

Peter J. Mogayzel, Jr., MD, PhD, MBA

Professor of PediatricsDirector, Cystic Fibrosis CenterJohns Hopkins University School of MedicineBaltimore, MD

Guest Faculty Disclosures:

Dr. Taylor-Cousar has indicated that, as faculty at an institution that is part of the CF TDN, she has been a site PI on studies for Bayer, Celtaxys, Proteostasis, and Vertex Pharmaceuticals Incorporated. She has also received consulting fees from Gilead Sciences, Inc and Santhera. She has done clinical trial consulting and/or advisory boards for Gilead Sciences, Inc., Proteostasis, Protalix, Santhera, and Vertex Pharmaceuticals Incorporated. She has received grant funding from Gilead Sciences, Inc.

Dr. Jia has indicated that she has no financial interests or relationships with a commercial entity.

Unlabeled/Unapproved Uses:

The authors have disclosed that they will be discussing the use of ivacaftor for rare mutations of CF. These methods have not been officially approved for this purpose.